EC approves Voxzogo, first treatment for children with achondroplasia

Following a positive opinion from the European Medicines Agency’s human health committee in June, the European Commission (EC) has rubber-stamped its marketing authorization for Voxzogo (vosoritide), a once daily injection to treat achondroplasia in children from the age of two until growth plates are closed, which occurs after puberty when children reach final adult height.

Developed by USA-based BioMarin Pharmaceutical (Nasdaq: BMRN), whose shares closed up 6% at 483.51 on the news Friday, Voxzogo is the first medicine to be approved to treat children with achondroplasia, the most common cause of dwarfism, in Europe. Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.

"Today represents an important milestone for the European achondroplasia community. For the first time medical professionals in Europe can offer a meaningful targeted therapeutic treatment option for children and families affected by achondroplasia," said Klaus Mohnike, Professor of Pediatrics at Magdeburg University Hospital in Germany and investigator for the Voxzogo clinical program.